Specialty Pipeline Update: May 2022

June 2, 2022
This monthly pipeline wrap-up provides a review of newly approved specialty drugs, recent specialty drug launches, new indications and news of note on specialty drugs in the approval process.

See separate article for pipeline information on traditional drugs.

New Drug Information

  • Camzyos™ (mavacamten, oral capsule): The U.S. Food and Drug Administration (FDA) approved Bristol-Myers Squibb (BMS)/MyoKardia’s Camzyos for the treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (HCM) to improve functional capacity and symptoms. This approval is based on data from the Phase 3 EXPLORER-HCM trial. At week 30, 37% of patients taking Camzyos achieved the composite primary endpoint, by achieving improvements of mixed venous oxygen tension (pVO2) by ≥1.5 mL/kg/min plus improvement in NYHA class by at least 1 or improvement of pVO2 by ≥3.0 mL/kg/min plus no worsening in NYHA class, versus 17% treated with placebo.1 Camzyos is the first FDA-approved allosteric and reversible inhibitor selective for cardiac myosin that targets the underlying pathophysiology of obstructive HCM. Beta blockers and nondihydropyridine calcium channel blockers are used off-label for the treatment of hypertrophic cardiomyopathy.2 Camzyos has launched and is available through a restricted program called the Camzyos REMS Program due to risk of heart failure due to systolic dysfunction. The wholesale acquisition cost (WAC) is $7,357 per 30 capsules.3
  • Cuvrior™ (trientine tetrahydrdrochloride, oral tablet): Orphalan’s Cuvrior has received FDA approval for the treatment of adult patients with stable Wilson’s disease who are de-coppered and tolerant to penicillamine. Wilson’s disease is a rare genetic disorder that prevents the body from removing extra copper, causing copper to build up in the liver, brain, eyes, and other organs. Without treatment, high copper levels can cause life-threatening organ damage.5 Penicillamine is approved as a first-line treatment option for Wilson’s disease, with about one third of patients developing intolerance while on therapy.6 The efficacy of Cuvrior was supported by the results of the Phase 3 trial, CHELATE, which met its primary efficacy endpoint by demonstrating that Cuvrior was non-inferior to penicillamine as measured by non-ceruloplasmin copper.5 Cuvrior was approved through the 505(b)(2) pathway using Valeant Pharmaceuticals’ Syprine® (trientine hydrochloride) as its reference product. A generic oral capsule formulation of trientine hydrochloride is available. At this time, Orphalan’s pricing and launch plans for Cuvrior have not been released.
  • Bortezomib™ (bortezomib, intravenous or subcutaneous injection): The FDA approved Hospira’s Bortezomib for the treatment of adult patients with multiple myeloma and for the treatment of adult patients with mantle cell lymphoma who have received at least one prior therapy. Bortezomib was approved through the 505(b)(2) pathway using Takeda’s Velcade®.6 Pricing and launch information are pending.
  • Radicava ORS™ (endaravone, oral solution): The FDA has approved Mitsubishi Tanabe Pharma America’s Radicava ORS oral suspension for the treatment of adults with amyotrophic lateral sclerosis (ALS). Radicava ORS is an orally administered version of Radicava®, which was originally approved in 2017 as an intravenous (IV) infusion to treat ALS, commonly referred to as Lou Gehrig’s disease. The effectiveness of Radicava ORS is based on a study that showed comparable levels of Radicava ORS in the bloodstream to the levels from the IV formulation of Radicava.7,8 The efficacy of Radicava for the treatment of ALS was previously demonstrated in a six-month clinical trial that served as the basis for approval in 2017. In that trial, 137 participants were randomized to receive Radicava or placebo.7,8 At week 24, individuals receiving Radicava declined less on a clinical assessment of daily functioning compared to those receiving placebo.7,8 The long-term safety and tolerability of Radicava ORS up to 96 weeks is currently being evaluated in an ongoing Phase 3 study.7,8 Radicava ORS 105mg/5 mL has launched with a WAC of $12,720 per 50 mL of solution.

 New Indications  

  • Ultomiris® (ravulizumab-cwvz): AstraZeneca announced the FDA approval of Ultomiris (ravulizumab-cwvz), for the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive.
  • Rinvoq® (upadacitinib): AbbVie announced the FDA approval of Rinvoq (upadacitinib), for the treatment of adults with active ankylosing spondylitis who have had an inadequate response or intolerance to one or more tumor necrosis factor (TNF) blockers.

 May News

  • “The evidence on outpatient treatments for COVID-19 must be viewed as highly sensitive to the evolving landscape of COVID-19 variants and vaccination status in the US,” said ICER President Steven Pearson, MD, MSc. “As indicated by the votes from the independent appraisal committee, the current evidence was judged more persuasive for Paxlovid and fluvoxamine than for molnupiravir, but clinical trials are ongoing for all three treatments.  At their current negotiated price (molnupiravir, and Paxlovid) or their generic market price (fluvoxamine), these drugs appear to have prices reasonably aligned with patient benefits. One of the key lessons to be learned from the development of these drugs is that the federal government’s advance market commitment mechanism was effective in reducing the financial uncertainty that could have deterred manufacturers from bringing a drug to market, and ultimately resulted in multiple drugs becoming available in a relatively short time at prices that were aligned with clinical benefit.  That experience has many lessons for the future of US policy in preparing for future pandemics.” A majority (11-2) found current evidence is notadequate to demonstrate a net health benefit when molnupiravir is compared to symptomatic care alone. All panelists (13-0) found that current evidence is adequate to demonstrate a net health benefit when Paxlovid is compared to symptomatic care alone. A slight majority (7-6) found that current evidence is adequate to demonstrate a net health benefit when fluvoxamine is compared to symptomatic care alone.”9
  • “The Institute for Clinical and Economic Review (ICER) today released a Draft Evidence Report assessing the comparative clinical effectiveness and value of betibeglogene autotemcel (Zynteglo/LentiGlobin, bluebird bio) for the treatment of beta thalassemia. Uncertainty remains about the degree of risk of beti-cel infusion in real-world practice, and there are known risks associated with myeloablative conditioning.6 Because of the questions about these risks and the durability of the clinical benefit, we judge that the evidence demonstrates that beti-cel is superior overall to the current standard of care, but the magnitude of that overall net health benefit is less certain, ranging between incremental to substantial (B+). Interestingly, we heard from many stakeholders, including patients with TDT and their families, that even if beti-cel proves to be a durable cure with an excellent safety profile, there are likely to be many patients who choose to continue their current management with transfusion and chelation. We performed cost-effectiveness analyses comparing beti-cel to the standard of care with co-base cases from the health care system and modified societal perspectives. Payment for beti-cel was assumed to be through five equal yearly payments in an outcomes-based contract that pays only for success, totaling $2.1 million for those patients who achieve and maintain transfusion independence through year five. Results were robust across numerous sensitivity and scenario analyses. The manufacturer has suggested publicly that beti-cel will be priced based on its clinical value to patients only rather than its ability to offset costs of current therapy.7 In a scenario analysis mirroring this approach, when half of the cost offsets from beti-cel treatment are retained by the health system, cost effectiveness estimates exceeded $200,000/evLY gained, demonstrating that cost offsets have a substantial impact on the cost effectiveness of this treatment. In summary, despite remaining uncertainties, the evidence suggests that beti-cel provides net health benefits to patients with TDT. Given the high annual costs of standard care, traditional cost-effectiveness modeling finds that this new treatment meets commonly accepted value thresholds at a cumulative price of $2.1 million if paid through an outcomes-based contract for patients with sustained transfusion independence.”10
  • “The FDA rebuked Pfizer CEO Albert Bourla’s proposed solution to reports that some patients experienced a relapse of COVID-19 symptoms after treatment with the company’s antiviral Paxlovid. After reports said some patients who took Paxlovid rebounded and started feeling symptoms again, the CEO toldBloomberg that patients can take another course, “like you do with antibiotics.” “Paxlovid does what it has to do: It reduces the viral load,” Bourla, Ph.D., told Bloomberg in an interview. “Then your body is supposed to do the job.” The FDA isn’t on board with the suggestion.“ There is no evidence of benefit at this time for a longer course of treatment … or repeating a treatment course of Paxlovid in patients with recurrent COVID-19 symptoms following completion of a treatment course,” John Farley, M.D., director of the Office of Infectious Diseases, said in a post.”11
  • “Digital therapeutics startup Pear Therapeutics brought in revenue of $2.7 million in the first quarter of 2022, up 108% from last quarter and up 618% year-over-year. Most of its revenue stems from sales of its three digital therapeutics: software-based treatments for substance use disorder, opioid use disorder and insomnia. Pear recently shared 12-month data on reSET-O, its digital therapeutic for opioid use disorder, and 24-month data on Somryst, its therapeutic for insomnia, showing a reduction in emergency department visits and hospitalization for patients who used these treatments. In summary, despite remaining uncertainties, the evidence suggests that beti-cel provides net health benefits to patients with TDT, and given the high annual costs of standard care, traditional cost-effectiveness modeling finds that this new treatment meets commonly accepted value thresholds at a cumulative price of $2.1 million if paid through an outcomes-based contract for patients with sustained transfusion independence.”12

*Specialty designation is not officially determined until FDA approval and launch.


  1. https://news.bms.com/news/details/2022/U.S.-Food-and-Drug-Administration-Approves-Camzyos-mavacamten-for-the-Treatment-of-Adults-With-Symptomatic-New-York-Heart-Association-Class-II-III-ObstructiveHypertrophic-Cardiomyopathy-HCM-to-Improve-Functional-Capacity-and-Symptoms/default.aspx
  2. https://www.uptodate.com/contents/hypertrophic-cardiomyopathy-medical-therapy-for-heart-failure?search=treatment%20of%20obstructive%20hypertrophic%20cardiomyopathy&source=search_result&selectedTitle=1~150&usage_type=default&display_rank=1#H5207476
  3. https://www.thepharmaletter.com/article/fda-green-light-for-bristol-myers-camzyos-in-ohcm
  4. https://www.orphalan.com/orphalan-announces-fda-approval-of-cuvrior/
  5. https://www.biospace.com/article/releases/fda-approves-orphalan-s-cuvrior-trientine-tetrahydrochloride-the-first-treatment-for-wilson-s-disease-in-over-five-decades/ https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/209191s000lbl.pdf
  6. https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/215446s000lbl.pdf
  7. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-oral-form-treatment-adults-amyotrophic-lateral-sclerosis-als
  8. https://icer.org/news-insights/press-releases/icer-publishes-final-evidence-report-and-policy-recommendations-on-outpatient-treatments-for-covid-19/
  9. https://icer.org/wp-content/uploads/2021/11/ICER_Beta-Thalassemia_Draft-Report_041322.pdf
  10. https://www.fiercepharma.com/pharma/fda-rebukes-pfizers-suggestion-take-more-paxlovid-if-covid-19-symptoms-return
  11. https://www.medtechdive.com/news/pear-revenue-doubles-q1-2022/623907/

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